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Pilot Study: A Parent-Infant Fragile X Intervention (PIXI)

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ClinicalTrials.gov Identifier: NCT03836300
Recruitment Status : Enrolling by invitation
First Posted : February 11, 2019
Last Update Posted : March 12, 2019
Sponsor:
Collaborator:
University of North Carolina, Chapel Hill
Information provided by (Responsible Party):
RTI International

Tracking Information
First Submitted Date  ICMJE February 7, 2019
First Posted Date  ICMJE February 11, 2019
Last Update Posted Date March 12, 2019
Actual Study Start Date  ICMJE November 30, 2018
Estimated Primary Completion Date December 1, 2022   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: March 8, 2019)
  • Social Validity and Acceptability [ Time Frame: Completion of Phase 1 (approximately six months of age) ]
    A social validity measure will be developed to inquire about family satisfaction with aspects of the intervention including curriculum, timing, goals targeted, and perceived effects of the intervention.
  • Social Validity and Acceptability [ Time Frame: Completion of Phase 2 (approximately twelve months of age) ]
    A social validity measure will be developed to inquire about family satisfaction with aspects of the intervention including curriculum, timing, goals targeted, and perceived effects of the intervention.
  • Caregiver Infant Interaction [ Time Frame: Completion of Phase 2 (approximately twelve months of age) ]
    The Manchester Assessment of Caregiver-Infant Interaction (MACI) is a global rating measure that covers the general features of parent, infant, and dyadic qualities of interaction. It is comprised of seven scales (two covering parent behavior [caregiver sensitive responsiveness, nondirectiveness], three on infant behavior [infant attentiveness to caregiver, affect, liveliness], and two focused on dyadic interaction [dyad mutuality, engagement intensity]) that cover broad aspects of interaction between a primary caregiver and their infant. Each scale is coded on a seven-point scale, with a higher rating indicating a more positive score. The MACI is a method of systematically evaluating qualitative or global aspects of behavioral interaction between a primary caregiver and their infant through rating their unstructured play from videotape.
  • Caregiver Infant Interaction [ Time Frame: Eighteen months of age ]
    The Manchester Assessment of Caregiver-Infant Interaction (MACI) is a global rating measure that covers the general features of parent, infant, and dyadic qualities of interaction. It is comprised of seven scales (two covering parent behavior [caregiver sensitive responsiveness, nondirectiveness], three on infant behavior [infant attentiveness to caregiver, affect, liveliness], and two focused on dyadic interaction [dyad mutuality, engagement intensity]) that cover broad aspects of interaction between a primary caregiver and their infant. Each scale is coded on a seven-point scale, with a higher rating indicating a more positive score. The MACI is a method of systematically evaluating qualitative or global aspects of behavioral interaction between a primary caregiver and their infant through rating their unstructured play from videotape.
Original Primary Outcome Measures  ICMJE
 (submitted: February 7, 2019)
  • Social Validity and Acceptability [ Time Frame: Completion of Phase 1 (approximately six months of age) ]
    A social validity measure will be developed to inquire about family satisfaction with aspects of the intervention including curriculum, timing, goals targeted, and perceived effects of the intervention.
  • Social Validity and Acceptability [ Time Frame: Completion of Phase 2 (approximately twelve months of age) ]
    A social validity measure will be developed to inquire about family satisfaction with aspects of the intervention including curriculum, timing, goals targeted, and perceived effects of the intervention.
  • Manchester Assessment of Caregiver Infant Interaction (MACI) [ Time Frame: Completion of Phase 2 (approximately twelve months of age) ]
    The MACI is a global rating measure, covering general features of parents, infant and dyadic qualities of interaction. It comprises of seven rating scales that covers broad aspects of interaction between a primary caregiver and their infant, each with a seven-point scale. It is a method of systematically evaluating qualitative or global aspects of behavioral interaction between a primary caregiver and infant through rating their unstructured play from videotape.
  • Manchester Assessment of Caregiver Infant Interaction (MACI) [ Time Frame: Eighteen months of age ]
    The MACI is a global rating measure, covering general features of parents, infant and dyadic qualities of interaction. It comprises of seven rating scales that covers broad aspects of interaction between a primary caregiver and their infant, each with a seven-point scale. It is a method of systematically evaluating qualitative or global aspects of behavioral interaction between a primary caregiver and infant through rating their unstructured play from videotape.
Change History Complete list of historical versions of study NCT03836300 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE
 (submitted: March 8, 2019)
  • Autism-associated Symptomology [ Time Frame: Completion of Phase 1 (approximately six months of age) ]
    The AOSI 18-item direct observational measure designed to detect and monitor putative signs of autism in infants aged 6-18 months
  • Autism-associated Symptomology [ Time Frame: Completion of Phase 2 (approximately twelve months of age) ]
    The Autism Observation Scale for Infants (AOSI) is an 18-item direct observational measure designed to detect and monitor putative signs of autism in infants aged 6-18 months. The scale results in an overall total score (sum of all items) ranging from 0-38, such that a higher score indicates potential symptomatology commonly associated with an autism spectrum disorder.
  • Autism Observation Scale for Infants (AOSI) [ Time Frame: Eighteen months of age ]
    The Autism Observation Scale for Infants (AOSI) is an 18-item direct observational measure designed to detect and monitor putative signs of autism in infants aged 6-18 months. The scale results in an overall total score (sum of all items) ranging from 0-38, such that a higher score indicates potential symptomatology commonly associated with an autism spectrum disorder.
  • Descriptive Statistics of Early Developmental Outcomes [ Time Frame: Completion of Phase 1 (approximately six months of age) ]
    The Bayley Scales of Infant and Toddler Development, Third Edition is a standardized assessment of early milestone-based development in young children, evaluating specific domains related to the development of infants from 1 to 42 months of age. Communication Scale: Scaled scores obtained on the receptive and expressive communication subtests are totaled to determine a standard composite score (range of 45-155), with a higher score indicating greater proficiency in that area. Motor Scale: Scaled scores obtained on the fine and gross motor subtests are totaled to determine a standard composite score (range of 45-155), with a higher score indicating greater proficiency in that area. Cognitive Scale: Measures constructs of early learning and cognition resulting in a scaled (range of 1-19) and standard composite score (range of 45-155), with a higher score indicating greater proficiency in that area.
  • Descriptive Statistics of Early Developmental Outcomes [ Time Frame: Completion of Phase 2 (approximately twelve months of age) ]
    The Bayley Scales of Infant and Toddler Development, Third Edition is a standardized assessment of early milestone-based development in young children, evaluating specific domains related to the development of infants from 1 to 42 months of age. Communication Scale: Scaled scores obtained on the receptive and expressive communication subtests are totaled to determine a standard composite score (range of 45-155), with a higher score indicating greater proficiency in that area. Motor Scale: Scaled scores obtained on the fine and gross motor subtests are totaled to determine a standard composite score (range of 45-155), with a higher score indicating greater proficiency in that area. Cognitive Scale: Measures constructs of early learning and cognition resulting in a scaled (range of 1-19) and standard composite score (range of 45-155), with a higher score indicating greater proficiency in that area.
  • Descriptive Statistics of Early Developmental Outcomes [ Time Frame: Eighteen months of age ]
    The Bayley Scales of Infant and Toddler Development, Third Edition is a standardized assessment of early milestone-based development in young children, evaluating specific domains related to the development of infants from 1 to 42 months of age. Communication Scale: Scaled scores obtained on the receptive and expressive communication subtests are totaled to determine a standard composite score (range of 45-155), with a higher score indicating greater proficiency in that area. Motor Scale: Scaled scores obtained on the fine and gross motor subtests are totaled to determine a standard composite score (range of 45-155), with a higher score indicating greater proficiency in that area. Cognitive Scale: Measures constructs of early learning and cognition resulting in a scaled (range of 1-19) and standard composite score (range of 45-155), with a higher score indicating greater proficiency in that area.
  • Descriptive Statistics of Early Developmental Outcomes [ Time Frame: Twenty-four months of age ]
    The Bayley Scales of Infant and Toddler Development, Third Edition is a standardized assessment of early milestone-based development in young children, evaluating specific domains related to the development of infants from 1 to 42 months of age. Communication Scale: Scaled scores obtained on the receptive and expressive communication subtests are totaled to determine a standard composite score (range of 45-155), with a higher score indicating greater proficiency in that area. Motor Scale: Scaled scores obtained on the fine and gross motor subtests are totaled to determine a standard composite score (range of 45-155), with a higher score indicating greater proficiency in that area. Cognitive Scale: Measures constructs of early learning and cognition resulting in a scaled (range of 1-19) and standard composite score (range of 45-155), with a higher score indicating greater proficiency in that area.
  • Descriptive Statistics of Early Developmental Outcomes [ Time Frame: Thirty-six months of age ]
    The Bayley Scales of Infant and Toddler Development, Third Edition is a standardized assessment of early milestone-based development in young children, evaluating specific domains related to the development of infants from 1 to 42 months of age. Communication Scale: Scaled scores obtained on the receptive and expressive communication subtests are totaled to determine a standard composite score (range of 45-155), with a higher score indicating greater proficiency in that area. Motor Scale: Scaled scores obtained on the fine and gross motor subtests are totaled to determine a standard composite score (range of 45-155), with a higher score indicating greater proficiency in that area. Cognitive Scale: Measures constructs of early learning and cognition resulting in a scaled (range of 1-19) and standard composite score (range of 45-155), with a higher score indicating greater proficiency in that area.
Original Secondary Outcome Measures  ICMJE
 (submitted: February 7, 2019)
  • Autism Observation Scale for Infants (AOSI) [ Time Frame: Completion of Phase 1 (approximately six months of age) ]
    The AOSI 18-item direct observational measure designed to detect and monitor putative signs of autism in infants aged 6-18 months
  • Autism Observation Scale for Infants (AOSI) [ Time Frame: Completion of Phase 2 (approximately twelve months of age) ]
    The AOSI 18-item direct observational measure designed to detect and monitor putative signs of autism in infants aged 6-18 months
  • Autism Observation Scale for Infants (AOSI) [ Time Frame: Eighteen months of age ]
    The AOSI 18-item direct observational measure designed to detect and monitor putative signs of autism in infants aged 6-18 months
  • Bayley Scales of Infant and Toddler Development-Third Edition (Bayley-III) [ Time Frame: Completion of Phase 1 (approximately six months of age) ]
    The Bayley-III is a standardized assessment of early milestone-based development in young children, evaluating five domains related to development of infants from 1 to 42 months of age: cognitive, language, motor, adaptive behavior, and social-emotional.
  • Bayley Scales of Infant and Toddler Development-Third Edition (Bayley-III) [ Time Frame: Completion of Phase 2 (approximately twelve months of age) ]
    The Bayley-III is a standardized assessment of early milestone-based development in young children, evaluating five domains related to development of infants from 1 to 42 months of age: cognitive, language, motor, adaptive behavior, and social-emotional.
  • Bayley Scales of Infant and Toddler Development-Third Edition (Bayley-III) [ Time Frame: Eighteen months of age ]
    The Bayley-III is a standardized assessment of early milestone-based development in young children, evaluating five domains related to development of infants from 1 to 42 months of age: cognitive, language, motor, adaptive behavior, and social-emotional.
  • Bayley Scales of Infant and Toddler Development-Third Edition (Bayley-III) [ Time Frame: Twenty-four months of age ]
    The Bayley-III is a standardized assessment of early milestone-based development in young children, evaluating five domains related to development of infants from 1 to 42 months of age: cognitive, language, motor, adaptive behavior, and social-emotional.
  • Bayley Scales of Infant and Toddler Development-Third Edition (Bayley-III) [ Time Frame: Thirty-six months of age ]
    The Bayley-III is a standardized assessment of early milestone-based development in young children, evaluating five domains related to development of infants from 1 to 42 months of age: cognitive, language, motor, adaptive behavior, and social-emotional.
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Pilot Study: A Parent-Infant Fragile X Intervention (PIXI)
Official Title  ICMJE Piloting an Early Intervention Program for Infants With Fragile X Syndrome
Brief Summary

The objective of this study is to develop and test, through an iterative process, an intervention to address and support the development of infants with a confirmed diagnosis of fragile X syndrome (FXS). This project will capitalize and expand upon existing empirically based interventions designed to improve outcomes for infants with suspected developmental and social delays.

Participants will be newborns with a confirmed diagnosis of FXS.

The intervention, called Parent-infant fragile X Intervention (PiXI) will consist of two phases. Phase 1 will include parent education about early infant development and FXS. Phase 2 includes direct parent coaching around parent-child interaction based on an empirically based parent-mediated early intervention and repeated comprehensive assessments of family and child functioning.

Detailed Description

Fragile X syndrome (FXS) is the leading known single-gene cause of autism spectrum disorder (ASD) and the most common inherited form of intellectual disability, resulting in significant functional impairments. Despite documentation that symptoms emerge in the first year of life), the average age of diagnosis is 3 years of age for males with FXS. Females with FXS are often diagnosed even later as they typically have a less severe phenotype. The delay of diagnosis can reduce timely access to interventions that could have significant consequences for the developmental trajectories of these children. Furthermore, the delay in diagnosis has significantly limited the ability of researchers to understand the natural progression of symptomology and thereby better address treatment options.

The primary goal of the proposed project is to develop and test, through an iterative process, an intervention to address and support the development of infants with a confirmed diagnosis of FXS who are identified at birth. PiXI aims to utilize the foundational knowledge available around the development of and early intervention for at-risk infants to both understand the needs of and provide intervention services for families of infants diagnosed pre-symptomatically with FXS.

The investigators aim to 1) develop PiXI with a pilot sample of families, 2) test the preliminary effects of PiXI on infant and parent outcomes. Specific infant outcomes include communication, motor, cognitive skills and autism spectrum disorder symptoms. Parental outcomes include parenting efficacy.

Study Type  ICMJE Interventional
Study Phase  ICMJE Not Applicable
Study Design  ICMJE Intervention Model: Single Group Assignment
Intervention Model Description:
The intervention, called Parent-infant fragile X Intervention (PiXI) will consist of two phases. Phase 1 will include parent education about early infant development and FXS through the use of Parents as Teachers procedures. Phase 2 includes direct parent coaching around parent-child interaction based on an empirically based parent-mediated early intervention, the Intervention within the British Autism study of Infant Siblings- Video-feedback Intervention to promote Positive parenting (i-BASIS-VIPP), and repeated comprehensive assessments of family and child functioning.
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Fragile X Syndrome
Intervention  ICMJE Behavioral: Parent-Infant fragile x Intervention (PIXI)

Phase 1 will include parent education about early infant development and FXS with support of Parents as Teachers programming.

Phase 2 includes direct parent coaching around parent-child interaction based on an empirically based parent-mediated early intervention, the Intervention within the British Autism study of Infant Siblings- Video-feedback Intervention to promote Positive parenting (i-BASIS-VIPP), and repeated comprehensive assessments of family and child functioning.

Study Arms  ICMJE Experimental: Infants with FXS and their parent/primary caregiver
Implement intervention in two phases
Intervention: Behavioral: Parent-Infant fragile x Intervention (PIXI)
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Enrolling by invitation
Estimated Enrollment  ICMJE
 (submitted: February 7, 2019)
120
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE December 1, 2022
Estimated Primary Completion Date December 1, 2022   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

  • Infants identified with FXS prior to 2 months of age with parents/caregivers who speak English

Exclusion Criteria:

-

Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE up to 6 Months   (Child)
Accepts Healthy Volunteers  ICMJE Yes
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03836300
Other Study ID Numbers  ICMJE 18-2079
Has Data Monitoring Committee No
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE Not Provided
Responsible Party RTI International
Study Sponsor  ICMJE RTI International
Collaborators  ICMJE University of North Carolina, Chapel Hill
Investigators  ICMJE
Principal Investigator: Anne Wheeler, PhD RTI International
PRS Account RTI International
Verification Date March 2019

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP